Zinc-Finger Nucleases for Somatic Gene Therapy: The Next Frontier
Zinc-finger nucleases (ZFNs) are a powerful tool that can be used to edit the human genome ad libitum. The technology has experienced remarkable development in the last few years with regard to both the target site specificity and the engineering platforms used to generate zinc-finger proteins. As a...
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| Veröffentlicht in: | Human gene therapy 2011-08, Vol.22 (8), p.925-933 |
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| creator | RAHMAN, Shamim H MAEDER, Morgan L KEITH JOUNG, J CATHOMEN, Toni |
| description | Zinc-finger nucleases (ZFNs) are a powerful tool that can be used to edit the human genome ad libitum. The technology has experienced remarkable development in the last few years with regard to both the target site specificity and the engineering platforms used to generate zinc-finger proteins. As a result, two phase I clinical trials aimed at knocking out the CCR5 receptor in T cells isolated from HIV patients to protect these lymphocytes from infection with the virus have been initiated. Moreover, ZFNs have been successfully employed to knockout or correct disease-related genes in human stem cells, including hematopoietic precursor cells and induced pluripotent stem cells. Targeted genome engineering approaches in multipotent and pluripotent stem cells hold great promise for future strategies geared toward correcting inborn mutations for personalized cell replacement therapies. This review describes how ZFNs have been applied to models of gene therapy, discusses the opportunities and the risks associated with this novel technology, and suggests future directions for their safe application in therapeutic genome engineering. |
| doi_str_mv | 10.1089/hum.2011.087 |
| format | Article |
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The technology has experienced remarkable development in the last few years with regard to both the target site specificity and the engineering platforms used to generate zinc-finger proteins. As a result, two phase I clinical trials aimed at knocking out the CCR5 receptor in T cells isolated from HIV patients to protect these lymphocytes from infection with the virus have been initiated. Moreover, ZFNs have been successfully employed to knockout or correct disease-related genes in human stem cells, including hematopoietic precursor cells and induced pluripotent stem cells. Targeted genome engineering approaches in multipotent and pluripotent stem cells hold great promise for future strategies geared toward correcting inborn mutations for personalized cell replacement therapies. This review describes how ZFNs have been applied to models of gene therapy, discusses the opportunities and the risks associated with this novel technology, and suggests future directions for their safe application in therapeutic genome engineering.</description><identifier>ISSN: 1043-0342</identifier><identifier>ISSN: 1557-7422</identifier><identifier>EISSN: 1557-7422</identifier><identifier>DOI: 10.1089/hum.2011.087</identifier><identifier>PMID: 21631241</identifier><identifier>CODEN: HGTHE3</identifier><language>eng</language><publisher>Larchmont, NY: Liebert</publisher><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy ; Applied cell therapy and gene therapy ; Biological and medical sciences ; Biotechnology ; CCR5 protein ; Endonucleases - genetics ; Fundamental and applied biological sciences. Psychology ; Gene therapy ; Genetic Engineering - methods ; Genetic Therapy - methods ; Genetic Therapy - trends ; Genome, Human ; Health aspects ; Health. Pharmaceutical industry ; HIV Infections - therapy ; Human immunodeficiency virus ; Humans ; Industrial applications and implications. Economical aspects ; Medical sciences ; Nucleases ; Physiological aspects ; Receptors, CCR5 - genetics ; Reviews ; Transfusions. Complications. Transfusion reactions. Cell and gene therapy ; Zinc finger proteins ; Zinc Fingers</subject><ispartof>Human gene therapy, 2011-08, Vol.22 (8), p.925-933</ispartof><rights>2015 INIST-CNRS</rights><rights>COPYRIGHT 2011 Mary Ann Liebert, Inc.</rights><rights>Copyright 2011, Mary Ann Liebert, Inc. 2011</rights><lds50>peer_reviewed</lds50><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed><citedby>FETCH-LOGICAL-c578t-fbfc8ac38ef8da4168458db66f911ed593c7f8921e7e2452f51e45770ad181573</citedby><cites>FETCH-LOGICAL-c578t-fbfc8ac38ef8da4168458db66f911ed593c7f8921e7e2452f51e45770ad181573</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><link.rule.ids>230,314,776,780,881,27901,27902</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=24484142$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/21631241$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>RAHMAN, Shamim H</creatorcontrib><creatorcontrib>MAEDER, Morgan L</creatorcontrib><creatorcontrib>KEITH JOUNG, J</creatorcontrib><creatorcontrib>CATHOMEN, Toni</creatorcontrib><title>Zinc-Finger Nucleases for Somatic Gene Therapy: The Next Frontier</title><title>Human gene therapy</title><addtitle>Hum Gene Ther</addtitle><description>Zinc-finger nucleases (ZFNs) are a powerful tool that can be used to edit the human genome ad libitum. The technology has experienced remarkable development in the last few years with regard to both the target site specificity and the engineering platforms used to generate zinc-finger proteins. As a result, two phase I clinical trials aimed at knocking out the CCR5 receptor in T cells isolated from HIV patients to protect these lymphocytes from infection with the virus have been initiated. Moreover, ZFNs have been successfully employed to knockout or correct disease-related genes in human stem cells, including hematopoietic precursor cells and induced pluripotent stem cells. Targeted genome engineering approaches in multipotent and pluripotent stem cells hold great promise for future strategies geared toward correcting inborn mutations for personalized cell replacement therapies. This review describes how ZFNs have been applied to models of gene therapy, discusses the opportunities and the risks associated with this novel technology, and suggests future directions for their safe application in therapeutic genome engineering.</description><subject>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</subject><subject>Applied cell therapy and gene therapy</subject><subject>Biological and medical sciences</subject><subject>Biotechnology</subject><subject>CCR5 protein</subject><subject>Endonucleases - genetics</subject><subject>Fundamental and applied biological sciences. Psychology</subject><subject>Gene therapy</subject><subject>Genetic Engineering - methods</subject><subject>Genetic Therapy - methods</subject><subject>Genetic Therapy - trends</subject><subject>Genome, Human</subject><subject>Health aspects</subject><subject>Health. Pharmaceutical industry</subject><subject>HIV Infections - therapy</subject><subject>Human immunodeficiency virus</subject><subject>Humans</subject><subject>Industrial applications and implications. Economical aspects</subject><subject>Medical sciences</subject><subject>Nucleases</subject><subject>Physiological aspects</subject><subject>Receptors, CCR5 - genetics</subject><subject>Reviews</subject><subject>Transfusions. Complications. Transfusion reactions. Cell and gene therapy</subject><subject>Zinc finger proteins</subject><subject>Zinc Fingers</subject><issn>1043-0342</issn><issn>1557-7422</issn><issn>1557-7422</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>2011</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkc9rFDEUgAdRbK3ePMuAiBdnzcuPScaDsBS3CqUerBcvIZt52Y3MJNtkVux_b4ZdqwVBcsgj-d57yfuq6jmQBRDVvd3uxwUlAAui5IPqFISQjeSUPiwx4awhjNOT6knO3wkBJlr5uDqh0DKgHE6r5TcfbLPyYYOpvtrbAU3GXLuY6i9xNJO39QUGrK-3mMzu9t0c1Ff4c6pXKYbJY3paPXJmyPjsuJ9VX1cfrs8_NpefLz6dLy8bK6SaGrd2VhnLFDrVGw6t4kL167Z1HQD2omNWOtVRQImUC-oEIBdSEtODAiHZWfX-UHe3X4_YWwxTMoPeJT-adKuj8fr-TfBbvYk_NAPRCcpLgdfHAine7DFPevTZ4jCYgHGfdUcoE5J07X9JpQQRZc6kkC8P5MYMqH1wsbS2M62XtBVKFjPz0xf_oMrqcfQ2BnS-nN9LeHNIsCnmnNDdfROInq3rYl3P1nWxXvAXf4_mDv6tuQCvjoDJ1gwumWB9_sNxrjhwyn4B_taywA</recordid><startdate>20110801</startdate><enddate>20110801</enddate><creator>RAHMAN, Shamim H</creator><creator>MAEDER, Morgan L</creator><creator>KEITH JOUNG, J</creator><creator>CATHOMEN, Toni</creator><general>Liebert</general><general>Mary Ann Liebert, Inc</general><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope><scope>7QO</scope><scope>7T7</scope><scope>7TM</scope><scope>8FD</scope><scope>C1K</scope><scope>FR3</scope><scope>P64</scope><scope>RC3</scope><scope>5PM</scope></search><sort><creationdate>20110801</creationdate><title>Zinc-Finger Nucleases for Somatic Gene Therapy: The Next Frontier</title><author>RAHMAN, Shamim H ; MAEDER, Morgan L ; KEITH JOUNG, J ; CATHOMEN, Toni</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c578t-fbfc8ac38ef8da4168458db66f911ed593c7f8921e7e2452f51e45770ad181573</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>2011</creationdate><topic>Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy</topic><topic>Applied cell therapy and gene therapy</topic><topic>Biological and medical sciences</topic><topic>Biotechnology</topic><topic>CCR5 protein</topic><topic>Endonucleases - genetics</topic><topic>Fundamental and applied biological sciences. Psychology</topic><topic>Gene therapy</topic><topic>Genetic Engineering - methods</topic><topic>Genetic Therapy - methods</topic><topic>Genetic Therapy - trends</topic><topic>Genome, Human</topic><topic>Health aspects</topic><topic>Health. Pharmaceutical industry</topic><topic>HIV Infections - therapy</topic><topic>Human immunodeficiency virus</topic><topic>Humans</topic><topic>Industrial applications and implications. Economical aspects</topic><topic>Medical sciences</topic><topic>Nucleases</topic><topic>Physiological aspects</topic><topic>Receptors, CCR5 - genetics</topic><topic>Reviews</topic><topic>Transfusions. Complications. Transfusion reactions. Cell and gene therapy</topic><topic>Zinc finger proteins</topic><topic>Zinc Fingers</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>RAHMAN, Shamim H</creatorcontrib><creatorcontrib>MAEDER, Morgan L</creatorcontrib><creatorcontrib>KEITH JOUNG, J</creatorcontrib><creatorcontrib>CATHOMEN, Toni</creatorcontrib><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><collection>Biotechnology Research Abstracts</collection><collection>Industrial and Applied Microbiology Abstracts (Microbiology A)</collection><collection>Nucleic Acids Abstracts</collection><collection>Technology Research Database</collection><collection>Environmental Sciences and Pollution Management</collection><collection>Engineering Research Database</collection><collection>Biotechnology and BioEngineering Abstracts</collection><collection>Genetics Abstracts</collection><collection>PubMed Central (Full Participant titles)</collection><jtitle>Human gene therapy</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>RAHMAN, Shamim H</au><au>MAEDER, Morgan L</au><au>KEITH JOUNG, J</au><au>CATHOMEN, Toni</au><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>Zinc-Finger Nucleases for Somatic Gene Therapy: The Next Frontier</atitle><jtitle>Human gene therapy</jtitle><addtitle>Hum Gene Ther</addtitle><date>2011-08-01</date><risdate>2011</risdate><volume>22</volume><issue>8</issue><spage>925</spage><epage>933</epage><pages>925-933</pages><issn>1043-0342</issn><issn>1557-7422</issn><eissn>1557-7422</eissn><coden>HGTHE3</coden><abstract>Zinc-finger nucleases (ZFNs) are a powerful tool that can be used to edit the human genome ad libitum. The technology has experienced remarkable development in the last few years with regard to both the target site specificity and the engineering platforms used to generate zinc-finger proteins. As a result, two phase I clinical trials aimed at knocking out the CCR5 receptor in T cells isolated from HIV patients to protect these lymphocytes from infection with the virus have been initiated. Moreover, ZFNs have been successfully employed to knockout or correct disease-related genes in human stem cells, including hematopoietic precursor cells and induced pluripotent stem cells. Targeted genome engineering approaches in multipotent and pluripotent stem cells hold great promise for future strategies geared toward correcting inborn mutations for personalized cell replacement therapies. This review describes how ZFNs have been applied to models of gene therapy, discusses the opportunities and the risks associated with this novel technology, and suggests future directions for their safe application in therapeutic genome engineering.</abstract><cop>Larchmont, NY</cop><pub>Liebert</pub><pmid>21631241</pmid><doi>10.1089/hum.2011.087</doi><tpages>9</tpages><oa>free_for_read</oa></addata></record> |
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| subjects | Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Applied cell therapy and gene therapy Biological and medical sciences Biotechnology CCR5 protein Endonucleases - genetics Fundamental and applied biological sciences. Psychology Gene therapy Genetic Engineering - methods Genetic Therapy - methods Genetic Therapy - trends Genome, Human Health aspects Health. Pharmaceutical industry HIV Infections - therapy Human immunodeficiency virus Humans Industrial applications and implications. Economical aspects Medical sciences Nucleases Physiological aspects Receptors, CCR5 - genetics Reviews Transfusions. Complications. Transfusion reactions. Cell and gene therapy Zinc finger proteins Zinc Fingers |
| title | Zinc-Finger Nucleases for Somatic Gene Therapy: The Next Frontier |
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